On 11/27/09, Theravance (NASDAQ:THRX) announced that it had received a Complete Response Letter (CRL) from the FDA for the telavancin New Drug Application (NDA) for the treatment of nosocomial pneumonia (NP). VIBATIV (telavancin) is approved in the US and Canada for the treatment of adult patients with complicated skin and skin structure infections (cSSSI) caused by susceptible Gram-positive bacteria. The CRL requested submission of additional data and analyses for the NP patient population to support an evaluation of all-cause mortality as the primary efficacy endpoint is necessary to demonstrate the safety and efficacy of telavancin.
The FDA also requested further rationale for the pooling of the data from the two ATTAIN studies, noting the need for a pediatric drug development plan for the NP indication and a customary safety update. THRX stated that the CRL requests were generally in line with its expectations and believes the requests can be addressed promptly. THRX believes that it has already collected the information and completed the analyses required to respond to the FDA. On 10/28/09, THRX and Astellas announced that Astellas Pharma Europe submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMEA) for telavancin, a bactericidal, once-daily injectable lipoglycopeptide antibiotic, for the treatment of nosocomial pneumonia, including ventilator-associated pneumonia, and complicated skin and soft tissue infections (cSSTI) in adults.
On 9/30/09, Merck KGaA (OTCPK:MKGAY) filed a NDA for FDA approval of its multiple sclerosis (MS) pill cladribine in the U.S. in a bid to bring the first oral treatment against the debilitating disease to market. The move follows a pivotal Phase 3 clinical trial, which showed in January that cladribine reduced the number of relapses per year in patients with some forms of MS by 58% compared with a placebo. On 11/30/09, the Company announced that the FDA rejected its application and reported that the next step is to request a meeting with the Agency and said that it is not in a position to discuss the details of the FDA letter.
On 11/30/09, BioMarin Pharma (NASDAQ:BMRN) announced that the FDA has granted orphan drug designation for 3,4-diaminopyridine (3,4-DAP), amifampridine phosphate, for the rare autoimmune disease Lambert Eaton Myasthenic Syndrome (LEMS). 3,4-DAP has previously received orphan drug designation in the E.U. Also, in October 2009, the Committee for Medicinal Products for Human Use of the European Medicines Evaluations Agency adopted a positive opinion recommending approval of amifampridine phosphate for LEMS. If approved by the European Commission, amifampridine phosphate will be the first approved treatment for LEMS, thereby conferring orphan drug protection and providing ten years of market exclusivity in Europe. BMRN expects to meet with the FDA in early 2010 to determine the necessary regulatory path for amifampridine phosphate in the U.S. and is also preparing to launch the product in Europe during 1Q10.
On 7/17/09, Cytori Therapeutics (NASDAQ:CYTX) announced that the FDA will regulate the Company's Celution 700 System as a medical device. This determination was made in response to the Company’s Request for Designation and clears the way for Cytori to compile and submit a marketing application to the FDA (either a 510k or PMA) for the Celution 700 System seeking marketing clearance as a medical device in aesthetic body contouring and/or filling of soft tissue voids. Cytori stated that it is preparing for the next steps in the process of working with the FDA to determine the specific device marketing application to submit, including whether clinical trials will be necessary for U.S. marketing clearance of the Celution 700 System.
Marketing applications for the Celution 700 device will be reviewed by the Agency’s Center for Biologics Evaluation and Research under the law applicable to medical devices. Clinical investigations, if required, would be conducted in accordance with the Investigational Device Exemption (IDE) regulations, not the Investigational New Drug (IND) regulations applicable to drugs and biologics. In late November, Cytori submitted its application for the Celution 700 AFT System to the FDA and the application is specific to aesthetic body contouring and/or filling of soft tissue voids through the processing and redistribution of autologous adipose tissue. The application could result in the clearance of the 700 AFT or a determination from the FDA that device-based clinical studies will be required.
Disclosure: Long CYTX